The purpose of this study is to investigate the safety and efficacy of rFIXFc in previously untreated patients (PUPs) in accordance with the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) guideline on clinical…
ID
Source
Brief title
Condition
- Coagulopathies and bleeding diatheses (excl thrombocytopenic)
- Blood and lymphatic system disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary objective of the study is to evaluate the safety of rFIXFc in
previously untreated subjects with severe hemophilia B.
Secondary outcome
The secondary objectives of the study are as follows:
To evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding
episodes in PUPs.
To evaluate rFIXFc consumption for prevention and treatment of bleeding
episodes in PUPs.
Background summary
The use of a prophylaxis regimen in young children starting prior to the onset
of frequent joint bleeding is currently the recommended standard of care in
hemophilia due to the demonstrated benefit on long-term outcomes replacement
therapies are limited by short elimination half-life requiring intravenous
administration up to 3 times per week.
Study objective
The purpose of this study is to investigate the safety and efficacy of rFIXFc
in previously untreated patients (PUPs) in accordance with the European
Medicines Agency Committee for Medicinal Products for Human Use (CHMP)
guideline on clinical investigation of recombinant and human plasma-derived
factor IX products.
Study design
An open-label, single-arm, multicenter study evaluating the safety and efficacy
of rFIXFc in previously untreated subjects with severe hemophilia B when used
according to local standard of care for implementation of a prophylaxis
regimen, including an optional preceding episodic (on-demand) treatment
regimen. Following the Baseline Visit, the Investigator has the option to treat
the subject episodically before initiating a prophylaxis regimen. The duration
of episodic treatment is at the Investigator*s discretion, in accordance with
local standard of care. The study will end when at least 40 subjects have
reached at least 100 EDs with rFIXFc. Surgery is allowed during the study.
Immune tolerance induction with rFIXFc is allowed during the study for those
subjects developing a positive inhibitor after exposure to rFIXFc study drug.
Adjustments to the dose and interval of rFIXFc can be made in this study based
on available pharmacokinetic (PK) data, subsequent FIX trough and peak levels,
level of physical activity, and bleeding pattern, in accordance with local
standards of care for a prophylactic regimen.
Intervention
Following the Baseline Visit, the Investigator has the option to treat the
subject episodically before initiating a prophylaxis regimen.
Episodic (on-demand) treatment is permitted; starting after the Baseline Visit
and lasting until a prophylactic regimen is initiated. Dosing will be
determined by the Investigator using Appendix A as a guideline. The duration of
episodic treatment should be based upon the Investigator*s treatment plan for
the subject in accordance with local standards.
The recommended initial prophylactic regimen is 50 IU/kg weekly. Adjustments to
the dose and dosing interval can be made based upon available incremental
recovery data (see Section 10.1.1), subsequent FIX trough and peak levels,
level of physical activity and bleeding pattern, in accordance with local
standards of care for a prophylactic regimen. Treatment will continue until the
subject has reached at least 100 EDs to rFIXFc study drug.
rFIXFc will be delivered by IV injection over 5 (±5) minutes; the rate of
administration should be determined by the subject*s comfort level.
Study burden and risks
rFIXFc can cause side effects and as with any new drug, there is a risk of rare
or previously unknown side effects, including cancer risks, allergy risks
and/or a chance that rFIXFc might interact with other drugs.
Second Avenue 225
Waltham MA 02451
US
Second Avenue 225
Waltham MA 02451
US
Listed location countries
Age
Inclusion criteria
1. Ability of the subject or their parent or legal guardian to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) or equivalent, and/or to provide assent in accordance with national and local regulations.;2. Male, age <18 years at the time of informed consent.;3. Weight *3.5 kg at the time of informed consent.;4. Severe hemophilia B defined as *2 IU/dL (*2%) endogenous FIX documented in the medical record or as tested during the Screening Period. Any subject who is enrolled based on results of the local laboratory must be withdrawn if the central laboratory screening results indicate a baseline FIX activity level >2% of normal.
Exclusion criteria
1. History of positive inhibitor testing. A prior history of inhibitors is defined based on a patient's historical positive inhibitor test using the local laboratory Bethesda value for a positive inhibitor test (i.e. equal to or above lower limit of detection).
2. History of hypersensitivity reactions associated with any rFIXFc administration.
3. Exposure to blood components or injection with a FIX concentrate (including plasma derived) other than rFIXc.
4. Injection with commercially available rFIXFc more than 28 days prior to Screening.
5. More than 3 injections of commercially available rFIXFc prior to confirmation of eligibility.
6. Other coagulation disorder(s) in addition to hemophilia B.
7. Any concurrent clinically significant major disease that, in the opinion of the Investigator, would make the subject unsuitable for enrollment (e.g., HIV infection with CD4 lymphocyte count <200 cells/µL or a viral load > 200 particles/µL, or any other known congenital or acquired immunodeficiency).
8. Current systemic treatment with chemotherapy and/or other immunosuppressant drugs. Use of steroids for treatment of asthma or management of acute allergic episodes or otherwise life-threatening episodes is allowed. Treatment in these circumstances should not exceed a 14-day duration.
9. Participation within the past 30 days in any other clinical study involving investigational treatment.
10. Current enrollment in any other clinical study involving investigational treatment.
11. Inability to comply with study requirements.
12. Other unspecified reasons that, in the opinion of the Investigator or Bioverativ, make the subject unsuitable for enrollment.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2013-003629-27-NL |
| ClinicalTrials.gov | NCT02234310 |
| CCMO | NL49000.042.14 |